Efficacy and Safety of Intranasal Cenegermin in Adult Participants With Non-Arteritic Anterior Ischemic Optic Neuropathy (NAION)

Randomized, Multicenter, Vehicle-Controlled, Double-Masked Phase 3 Study to Evaluate the Efficacy and Safety of Intranasal Cenegermin (Recombinant Human Nerve Growth Factor [rhNGF]) in Adult Participants With Non-Arteritic Anterior Ischemic Optic Neuropathy (NAION)

This is a phase 3, randomized, multicenter, vehicle-controlled, double-masked study to evaluate the efficacy and safety of intranasal cenegermin compared with vehicle control in adult participants with NAION. Approximately 272 participants who meet all eligibility criteria will be randomly assigned in a 1:1 ratio to receive either cenegermin treatment (Group 1) or the vehicle control (Group 2).

Study Overview

• Status: Recruiting
• Conditions: Non-Arteritic Anterior Ischemic Optic Neuropathy
• Intervention / Treatment: Drug: Cenegermin; Other: Vehicle

• Study Type: Interventional
• Enrollment (Estimated): 272
• Phase: Phase 3

Contacts and Locations

• Study Contact: Name: Dompé farmaceutici S.p.A. Via Santa Lucia, 6, 20122 Milan (MI); Phone Number: +39 02 583 831

Study Locations

• United States
  • California
    • Los Angeles, California, United States, 90048
      • Recruiting
      • Neuro Eye Orbit Institute
    • Los Angeles, California, United States, 90095
      • Not yet recruiting
      • David Geffen School of Medicine at UCLA - Ophthalmology
    • Newport Beach, California, United States, 92660
      • Not yet recruiting
      • California Orbital Consultants
  • Colorado
    • Aurora, Colorado, United States, 80045
      • Recruiting
      • University of Colorado Anschutz Medical Campus
  • Florida
    • Miami, Florida, United States, 33136
      • Not yet recruiting
      • Bascom Palmer Eye Institute, University of Miami School of Medicine
    • Sarasota, Florida, United States, 34239
      • Not yet recruiting
      • Sarasota Retina Institute - Research
  • Illinois
    • Chicago, Illinois, United States, 60611
      • Not yet recruiting
      • Northwestern University - Ophthalmology
    • Chicago, Illinois, United States, 60612
      • Not yet recruiting
      • University of Illinois at Chicago Eye and Ear Infirmary - Ophthalmology
  • Kentucky
    • Lexington, Kentucky, United States, 40508
      • Not yet recruiting
      • Univ. Of Kentucky Clinic Dept of Ophthalmology & Vision Science
  • Maryland
    • Baltimore, Maryland, United States, 21287
      • Not yet recruiting
      • The Johns Hopkins Hospital (JHH)
  • Massachusetts
    • Boston, Massachusetts, United States, 02116
      • Not yet recruiting
      • Tufts Medical Center - Neurology
  • Michigan
    • Ann Arbor, Michigan, United States, 48105
      • Recruiting
      • University of Michigan Health - Kellogg Eye Center
    • Grand Rapids, Michigan, United States, 49503
      • Not yet recruiting
      • Trinity Health Grand Rapids
  • Minnesota
    • Minneapolis, Minnesota, United States, 55455
      • Not yet recruiting
      • University of Minnesota, Dept of Ophthalmology
    • Rochester, Minnesota, United States, 55905
      • Recruiting
      • Mayo Clinic - Department of Ophthalmology
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19104
      • Recruiting
      • University of Pennsylvania - Ophthalmology
  • South Carolina
    • Charleston, South Carolina, United States, 29414
      • Recruiting
      • Retina Consultants of South Carolina, Charleston Neuroscience Institute- West Ashley (RCA)
  • Texas
    • Houston, Texas, United States, 77074
      • Recruiting
      • Neuro Eye Clinical Trials

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Key Inclusion Criteria:

1. A clinical diagnosis of unilateral NAION in the study eye with symptom onset within 14 days prior to the planned date for first dose administration.
2. A BCVA score in the study eye of ≥ 15 letters and ≤ 65 letters measured using the ETDRS chart.
3. Sufficiently clear ocular media and adequate pupil dilation to enable assessment of the optic nerve and retina in both eyes.

Key Exclusion Criteria:

1. Bilateral NAION or sequential NAION with fellow eye involvement within 6 weeks of study eye involvement.
2. Clinical evidence of temporal arteritis (giant cell arteritis) signs or symptoms.
3. Abnormal laboratory findings suggestive of temporal arteritis (giant cell arteritis), in the absence of a known acute cause
4. Pain with eye movement
5. Intraocular pressure (IOP) greater than 25 mmHg in the study eye or history of glaucoma in the study eye.
6. Intermediate age-related macular degeneration (AMD) with subfoveal drusen, exudative AMD, or geographic atrophy in the study eye.

Note: Additional inclusion/exclusion criteria apply, as defined in the protocol.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Triple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Cenegermin; Participants will be administered with cenegermin spray in each nostril.	Drug: Cenegermin; Cenegermin is administered intranasally.
Placebo Comparator: Vehicle; Participants will be administered with vehicle spray in each nostril.	Other: Vehicle; Vehicle spray is administered intranasally.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Achievement of ≥ 15 letter increase in Best Corrected Visual Acuity (BCVA), assessed in each individual participant, measured using the Early Treatment Diabetic Retinopathy Study (ETDRS) chart	Improvement in visual acuity defined as a >=15 letter increase in BCVA using ETDRS chart.	Baseline to Week 24

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change from baseline in visual field mean sensitivity in decibels	Change from baseline in visual field mean sensitivity in decibels will be analyzed	Baseline through Week 24
Change from baseline in BCVA as measured by the ETDRS chart		Baseline through Week 24
Change from baseline in visual field mean sensitivity, in a prespecified region consisting of at least 5 separate loci on visual field testing	Change from baseline in mean sensitivity at week 24 in loci prespecified at baseline will be analyzed.	Baseline through Week 24
Change from baseline in ganglion cell layer-inner plexiform layer (GCL-IPL) thickness (micrometer [μm]) on optical coherence tomography (OCT).	Change from baseline in GCL-IPL thickness on optical coherence tomography will be analyzed.	Baseline through Week 24
Incidence of ocular and non-ocular adverse events	Number of participants with ocular and non-ocular adverse events will be assessed.	Through Week 24

Collaborators and Investigators

• Sponsor: Dompé Farmaceutici S.p.A

Study record dates

Study Major Dates

• Study Start (Actual): July 1, 2026
• Primary Completion (Estimated): September 1, 2027
• Study Completion (Estimated): September 1, 2027

Study Registration Dates

• First Submitted: March 2, 2026
• First Submitted That Met QC Criteria: March 2, 2026
• First Posted (Actual): March 6, 2026

Study Record Updates

• Last Update Posted (Actual): July 15, 2026
• Last Update Submitted That Met QC Criteria: July 13, 2026
• Last Verified: July 1, 2026

More Information

Terms related to this study

• Keywords: cenegermin; Non-Arteritic Anterior Ischemic Optic Neuropathy; NAION; Intranasal; Optic neuropathy; Acute optic neuropathy
• Additional Relevant MeSH Terms: Nervous System Diseases; Vascular Diseases; Cardiovascular Diseases; Eye Diseases; Cranial Nerve Diseases; Optic Nerve Diseases; Optic Neuropathy, Ischemic; cenegermin
• Other Study ID Numbers: NGF-NAION-301

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: Yes